--Findings support the versatility of Capricor’s exosome technology, enabling efficient and scalable loading of siRNAs and PMOs to support future clinical development-- SAN DIEGO, Nov. 24, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc., (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics, today announced new data describing a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into ...

Core Insights - Capricor Therapeutics is approaching a critical phase in its development with the upcoming topline results from the HOPE-3 Phase 3 trial of Deramiocel for Duchenne muscular dystrophy (DMD) [2][4][5] - The company has prepared for a potential commercial launch of Deramiocel in 2026, contingent on the trial results and regulatory approval [4][5] - Financial results indicate a significant increase in operating expenses and a net loss for the third quarter of 2025 compared to the same period in 2024 [9][10] Company Updates - The topline results from the HOPE-3 Phase 3 study, which includes 105 participants, are expected in Q4 2025 [4][5] - Following a recent Type A meeting with the FDA, Capricor plans to resubmit its Biologics License Application (BLA) using the HOPE-3 results to address previous concerns raised in a Complete Response Letter [5] - The company has completed its FDA Pre-License Inspection for its commercial manufacturing facility, which is now operational and ready for initial product launch [5] Financial Performance - As of September 30, 2025, Capricor reported cash, cash equivalents, and marketable securities totaling approximately $98.6 million, down from $151.5 million at the end of 2024 [7][25] - Revenues for Q3 2025 were $0, a decrease from approximately $2.3 million in Q3 2024, with total revenues for the first nine months of 2025 also at $0 compared to approximately $11.1 million in the same period of 2024 [8] - Total operating expenses for Q3 2025 were approximately $26.3 million, up from approximately $15.3 million in Q3 2024, leading to a net loss of approximately $24.6 million for Q3 2025 compared to a net loss of approximately $12.6 million in Q3 2024 [9][10] Upcoming Developments - Capricor is preparing for a commercial launch of Deramiocel in 2026, pending the results of the HOPE-3 trial [4][5] - The company is also advancing its StealthX™ exosome-based vaccine platform, with initial data from a Phase 1 clinical trial expected in Q1 2026 [4][6]

Core Insights - Capricor Therapeutics has published a study on Deramiocel's anti-fibrotic activity, highlighting a validated potency assay that supports quality control in late-stage development for Duchenne muscular dystrophy (DMD) [1][4] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapies for rare diseases, with Deramiocel as its lead investigational product [1][10] - The company has received multiple designations for Deramiocel, including Orphan Drug Designation from the U.S. FDA and EMA, and Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. [7] Research Findings - The publication details a novel in-vitro potency assay that characterizes the anti-fibrotic mechanism of action of cardiosphere-derived cells (CDCs) in Deramiocel, showing that CDCs can suppress collagen I and III gene expression in human fibroblasts [2][3] - Consistent findings across over one hundred manufacturing lots of Deramiocel demonstrate its anti-fibrotic mechanism, aligning with in vivo studies and clinical data showing stabilization of cardiac function in DMD patients [3] Clinical Development - Deramiocel is currently being evaluated in the Phase 3 HOPE-3 trial, with topline data expected in mid-fourth quarter of 2025 [4][8] - The HOPE-3 trial is a multi-center, randomized, double-blind, placebo-controlled study involving 105 eligible subjects [8][9] Mechanism of Action - Deramiocel consists of allogeneic CDCs that exert immunomodulatory and anti-fibrotic actions, preserving cardiac and skeletal muscle function in muscular dystrophies [6][10] - CDCs secrete exosomes that target macrophages, promoting a healing phenotype rather than a pro-inflammatory one [6] Educational Initiatives - To complement the publication, Capricor has released an educational video explaining the mechanism of action of Deramiocel [5]

Core Insights - Capricor Therapeutics will release its financial results for Q3 2025 on November 10, 2025, followed by a conference call at 4:30 p.m. ET [1] - The company is focused on developing cell and exosome-based therapeutics for rare diseases, with its lead product candidate being Deramiocel for Duchenne muscular dystrophy (DMD) [2] Company Overview - Capricor Therapeutics is dedicated to advancing transformative therapies, particularly through its lead candidate Deramiocel, which is in late-stage clinical development for DMD [2] - Deramiocel has shown potent immunomodulatory and anti-fibrotic effects in preserving cardiac and skeletal muscle function in DMD patients [2] - The company is also utilizing its proprietary StealthX™ platform for the targeted delivery of therapeutics, with applications in vaccinology and treatment of various diseases [2] Recent Developments - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [5]

Company Overview - Capricor Therapeutics (CAPR) shares increased by 5.3% to close at $6.52, with trading volume significantly higher than usual, contrasting with a 1% loss over the past four weeks [1][2]. Regulatory Update - The rise in CAPR shares is linked to a positive regulatory update regarding the Deramiocel program for Duchenne muscular dystrophy, following a Type A meeting with the FDA that provided guidance for a potential Biologics License Application [2]. Financial Expectations - The company is projected to report a quarterly loss of $0.54 per share, reflecting a year-over-year decline of 42.1%, while revenues are expected to reach $2.4 million, marking a 6.2% increase from the previous year [3]. Earnings Estimate Trends - The consensus EPS estimate for Capricor has remained stable over the last 30 days, indicating that stock price movements typically do not sustain upward trends without changes in earnings estimate revisions [4]. Industry Context - Capricor is part of the Zacks Medical - Products industry, where another company, biote Corp. (BTMD), experienced a 0.3% decline to $2.97, with a return of -11.3% over the past month [5].

Core Insights - Capricor Therapeutics announced a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), following a Type A meeting with the FDA after receiving a Complete Response Letter (CRL) in July 2025 [1][2][3] Regulatory Update - The Type A meeting aimed to establish a path toward potential approval of Deramiocel, with key outcomes providing clarity on the regulatory strategy and the opportunity to use HOPE-3 data for approval if it meets regulatory requirements [2][3] - The FDA has aligned with Capricor on endpoints for the HOPE-3 pivotal trial, which is expected to support the BLA resubmission [7][8] Clinical Trials - The HOPE-3 trial is designed to validate findings from previous studies (HOPE-2 and HOPE-2-OLE) that demonstrated significant benefits in cardiac and skeletal muscle function [3][8] - The trial consists of two cohorts evaluating the safety and efficacy of Deramiocel in DMD participants, with 105 subjects enrolled [11] Financial Position - Capricor maintains a strong financial position to support the advancement of Deramiocel through regulatory review and potential launch [4] Product Information - Deramiocel (CAP-1002) is composed of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic actions in preserving muscle function in muscular dystrophies [9][12] - The product has received multiple designations, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation, which may qualify Capricor for a Priority Review Voucher upon approval [10]

Core Insights - Capricor Therapeutics has initiated a Phase 1 clinical trial for its StealthX™ exosome-based vaccine, marking a significant milestone in vaccine development [1][2] - The trial is funded by the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) and follows FDA clearance of the Investigational New Drug (IND) application [1][2] - The StealthX™ platform aims to provide an alternative to mRNA vaccines, utilizing a more natural delivery system without adjuvants [2] Company Overview - Capricor Therapeutics is focused on developing transformative cell and exosome-based therapeutics for rare diseases, with its lead product candidate being Deramiocel, a therapy for Duchenne Muscular Dystrophy (DMD) [5][8] - The company is leveraging its proprietary StealthX™ platform for vaccine development and targeted delivery of therapeutics [5] Clinical Trial Details - The Phase 1 trial includes four dosing arms, initially targeting the spike protein of SARS-CoV-2, with plans for an additional arm focusing on the nucleocapsid protein pending FDA clearance [2] - Initial data from the trial is expected in the first quarter of 2026 [2] Strategic Collaborations - Capricor is collaborating with the NIH on the trial and has held discussions with the FDA regarding its Deramiocel program for DMD [2][8] - The company has entered into an agreement with Nippon Shinyaku Co., Ltd. for the exclusive commercialization and distribution of Deramiocel in the U.S. and Japan, subject to regulatory approval [8]

Core Insights - Capricor Therapeutics is focused on developing therapies for Duchenne cardiomyopathy and has faced regulatory challenges but is working on a clear path forward for approval [2][4] - The company has made significant progress with its exosome platform, StealthX™, which has received FDA clearance for clinical trials [4][6] - Financial results indicate a net loss for the second quarter of 2025, with revenues dropping to zero compared to the previous year [8][10] Corporate Update - Capricor aims to resubmit its Biologics License Application (BLA) for Deramiocel based on existing data, with additional data from the HOPE-3 trial expected in Q4 2025 [4][5] - The FDA has accepted all observations from the Pre-License Inspection, marking a milestone in regulatory progress [4][5] - The company is preparing for a Type A meeting with the FDA to discuss the approval pathway [4][5] Financial Performance - Cash position as of June 30, 2025, was approximately $122.8 million, down from $151.5 million at the end of 2024 [7][26] - Revenues for Q2 2025 were $0, a decrease from approximately $4.0 million in Q2 2024, and for the first half of 2025, revenues were also $0 compared to approximately $8.9 million in the first half of 2024 [8][10] - Total operating expenses for Q2 2025 were approximately $27.7 million, up from $15.6 million in Q2 2024 [9][10] Clinical Development - The HOPE-3 Phase 3 clinical trial is ongoing, with topline data expected in Q4 2025 [5][6] - The FDA has cleared the IND for the StealthX™ exosome-based vaccine, marking its first clinical entry [4][6] - Orphan Drug Designation has been granted for Deramiocel in Becker muscular dystrophy, expanding its therapeutic strategy [5][6]

Core Viewpoint - Capricor Therapeutics is advancing its lead cell therapy candidate, Deramiocel, for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) and has scheduled a Type A meeting with the FDA to discuss its regulatory path for the Biologics License Application (BLA) [1][4][7] Financial Results and Corporate Update - The release of Capricor's financial results for the second quarter ended June 30, 2025, has been rescheduled to August 11, 2025, after market close [2][3] - A conference call and webcast will be held on the same day at 4:30 p.m. ET to review the financial results and provide a corporate update [2][3] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel being a key product candidate showing immunomodulatory and anti-fibrotic effects in muscular dystrophies [4] - The company is also utilizing its proprietary StealthX™ platform for preclinical development in areas such as vaccinology and targeted delivery of therapeutics [4] Commercialization Agreement - Capricor has entered into an exclusive agreement with Nippon Shinyaku Co., Ltd. for the commercialization and distribution of Deramiocel for DMD in the United States and Japan, pending regulatory approval [7]

Core Insights - Capricor Therapeutics is hosting a webinar on July 29, 2025, to discuss the status of its Biologics License Application (BLA) for Deramiocel and provide information on cardiomyopathy in Duchenne Muscular Dystrophy (DMD) [1][2] - Deramiocel is an allogeneic cardiac-derived cell therapy currently in late-stage development for DMD, demonstrating potent immunomodulatory and anti-fibrotic actions [3][5] - Capricor has entered an exclusive commercialization agreement for Deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [5] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with a commitment to innovative treatments [3] - The company utilizes its proprietary StealthX™ platform for preclinical development in various therapeutic areas, including vaccinology and targeted delivery [3] Webinar Details - The webinar will include a review of the BLA status for Deramiocel and a Q&A session with the community [1] - A replay of the webinar will be available on the Parent Project Muscular Dystrophy (PPMD) website [2]